FDA批準復發(fā)難治性套細胞淋巴瘤的治療藥，臨床試驗結果顯示腫瘤縮小的有效率很高。

       For Immediate Release:

       November 14, 2019

       Today, the U.S. Food and Drug Administration granted accelerated approval to Brukinsa (zanubrutinib) capsules for the treatment of adult patients with mantle cell lymphoma who have received at least one prior therapy.

       今天，美國食品和藥物管理局批準Brukinsa（Zanubrutinib）用于治療至少接受過一次治療的成人套細胞淋巴瘤患者。

       “Mantle cell lymphoma usually responds well to initial treatment, but eventually returns or stops responding, and the cancer cells continue to grow. This is a life-threatening condition,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Clinical trials showed that 84% of patients saw tumor shrinkage with this therapy. For patients whose disease relapses or becomes refractory, secondary therapies may be successful in providing another remission, and today’s approval will provide patients with another treatment option.”

       FDA腫瘤中心主任、FDA藥物評價和研究中心腫瘤疾病辦公室代理主任、醫(yī)學博士Richard Pazdur表明：“套細胞淋巴瘤通常對最初的治療反應良好，但最終會減緩或停止作用，癌細胞繼續(xù)生長，這是一種危及生命的疾病。臨床試驗表明，84%的患者認為這種療法會導致腫瘤縮小。對于疾病復發(fā)或變得難治的患者，二次治療可能會成功地提供另一種緩解，今天的批準將為患者提供另一種治療選擇。”

       Mantle cell lymphoma is a type of non-Hodgkin’s lymphoma representing 3-10% of all non-Hodgkin’s lymphomas in the United States. By the time it is diagnosed, mantle cell lymphoma has usually spread to the lymph nodes, bone marrow and other organs. In relapsed lymphoma, the disease reappears or grows again after a period of remission, while in refractory lymphoma, the disease does not respond to treatment or responds only briefly.

       套細胞淋巴瘤是一種非霍奇金淋巴瘤，占美國非霍奇金淋巴瘤的3-10%。到確診時，套細胞淋巴瘤通常已經擴散到淋巴結、骨髓和其他器官。在復發(fā)性淋巴瘤中，該病在一段時間的緩解后再次出現(xiàn)或生長，而在難治性淋巴瘤中，該病對治療無反應或僅短暫反應。

       A single-arm clinical trial of Brukinsa included 86 patients with mantle cell lymphoma who had received at least one prior treatment. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 84% of patients had tumor shrinkage with a median duration of response (time between the initial response to therapy and subsequent disease progression or relapse) of 19.5 months. This trial was supported by an additional single-arm trial that included 32 patients, in which 84% of patients had tumor shrinkage with a median duration of response of 18.5 months.

       Brukinsa的隨訪臨床試驗包括86例經至少一次治療的套細胞淋巴瘤患者。該試驗測量了多少患者在治療后腫瘤完全或部分縮?。傮w響應率）。在試驗中，84%的患者腫瘤縮小，反應持續(xù)時間中位數（從治療的最初反應到隨后的疾病進展或復發(fā)之間的時間）為19.5個月。這項試驗得到了另一項包括32名患者的隨訪試驗的支持，其中84%的患者腫瘤縮小，緩解時間中位數為18.5個月。

       Common side effects for patients taking Brukinsa were decreased neutrophil count (white blood cells that fight against infection), decreased platelet count (a component of blood whose function is to react to bleeding from blood vessel injury by clumping, initiating a blood clot), upper respiratory tract infection, decreased white blood cell count, decreased hemoglobin (oxygen-carrying protein in red blood cells), rash, bruising, diarrhea and cough. During treatment, patients should be monitored for hemorrhage (bleeding), signs and symptoms of infection, cytopenias (decreased complete blood counts) and cardiac arrhythmias (irregular, rapid heart rate and abnormality in beating of the heart). Patients are advised to use sun protection if taking this therapy because there is a risk of other malignancies occurring including skin cancers. The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Brukinsa. Women who are pregnant or breastfeeding should not take Brukinsa because it may cause harm to a developing fetus or newborn baby.

       服用Brukinsa的患者的常見副作用是中性粒細胞數（對抗感染的白細胞）、血小板數（血液的一種成分，其功能是對血管損傷引起的出血作出反應，形成血栓）、上呼吸道感染、白細胞數下降，血紅蛋白（紅細胞中的含氧蛋白）、皮疹、瘀傷、腹瀉和咳嗽減少。在治療過程中，應監(jiān)測患者的出血、感染癥狀和體征、細胞減少（全血減少）和心律失常（心率不規(guī)則、快速和心跳異常）。如果患者接受這種療法，建議使用防曬霜，因為有可能發(fā)生其他惡性腫瘤，包括皮膚癌。FDA建議衛(wèi)生保健專業(yè)人員告訴育齡女性和有生殖潛能的女性伴侶的男性在治療Brukinsa期間使用有效的避孕措施。孕婦或哺乳期婦女不應服用Brukinsa，因為它可能對發(fā)育中的胎兒或新生兒造成傷害。

       Brukinsa was granted Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients. Further clinical trials may be required to verify and describe Brukinsa’s clinical benefit.

       Brukinsa獲得了加速批準，這使得FDA能夠批準重要情況下的藥物，以滿足未滿足的醫(yī)療需求，其結果有可能合理預測患者的臨床益處。可能需要進一步的臨床試驗來驗證和描述Brukinsa的臨床益處。

       The FDA granted this application Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Brukinsa also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted approval of Brukinsa to BeiGene USA Inc.

       FDA批準了這一突破性的治療方案，加速了治療嚴重疾病藥物的開發(fā)和審查，初步臨床證據表明，該藥物可能比現(xiàn)有療法有實質性的改善。Brukinsa還獲得了罕見病用藥指定，這為幫助和鼓勵開發(fā)治療罕見疾病的藥物提供了獎勵。FDA批準了百濟神州美國分公司生產Brukinsa。

       The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

       FDA是美國衛(wèi)生和公共服務部的一個機構，通過確保人用、獸藥、**和其他生物制品以及醫(yī)療器械的安全性、有效性和安全性來保護公眾健康。該機構還負責美國食品供應、化妝品、膳食補充劑、釋放電子輻射的產品的安全和保障，以及管制煙草產品。